FMT for Autism

Overseen By Sonia Michail, MD

Age: < 18

Sex: Any

Trial Phase: Phase 1

Sponsor: Children's Hospital Los Angeles

Disqualifiers: Renal dysfunction, Liver dysfunction, Immunodeficiency, others

Compensation: Varies

Number of Visits: 8

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether fecal microbiota transplantation (FMT) can help children and young adults with Autism Spectrum Disorder (ASD). FMT transfers healthy bacteria from a donor's stool into the intestines, potentially improving gut health and affecting ASD symptoms. The study includes two groups: one receiving FMT and the other a placebo. Children aged 5-17 with an ASD diagnosis and specific gastrointestinal (GI) problems may be suitable candidates. Participants will attend several visits over 24 weeks to monitor changes. As a Phase 1 trial, this research aims to understand how FMT works in people, offering participants the opportunity to be among the first to receive this innovative treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that fecal microbiota transplantation (FMT) is generally safe for patients. Studies have found that FMT can improve stomach and behavior issues in children with autism spectrum disorder (ASD). Although the treatment is considered safe, some individuals might experience mild side effects such as bloating, gas, or diarrhea. These effects are usually temporary and not serious. As this study is in an early stage, it primarily focuses on the safety of FMT. While the results are promising, further research is needed to fully understand the long-term safety and effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Autism?

Unlike the standard treatments for autism, which often involve behavioral therapy or medications that target symptoms, FMT (Fecal Microbial Transplant) introduces a completely different approach by focusing on the gut microbiome. Researchers are excited because FMT involves transferring healthy donor stool into the patient's jejunum, potentially restoring gut flora balance. This method is based on emerging evidence linking gut health to neurological conditions, offering a novel angle for autism treatment that could lead to improvements in symptoms by addressing underlying causes rather than just managing them.

What evidence suggests that FMT might be an effective treatment for Autism?

Research has shown that fecal microbiota transplantation (FMT) might improve symptoms in children with Autism Spectrum Disorder (ASD). In this trial, one group of participants will receive FMT using healthy donor stool. Studies have found that this can enhance gut health and reduce core ASD symptoms. The treatment introduces healthy gut bacteria, potentially balancing the gut and alleviating symptoms. Some early research also suggests FMT could help with anxiety often associated with ASD. Although more research is needed, these early results are promising for using FMT to manage ASD symptoms. ^{1 2 4 5 6}

Who Is on the Research Team?

Sonia Michail, MD

Principal Investigator

Children's Hospital Los Angeles

Pat Levitt, Ph.D

Principal Investigator

Children's Hospital Los Angeles

Are You a Good Fit for This Trial?

This trial is for children aged 5-17 with Autism Spectrum Disorder (ASD) who have gastrointestinal symptoms. They must be diagnosed using specific autism and GI symptom assessments, need an upper GI endoscopy, and not have any severe sensory or motor problems, genetic syndromic disorders, significant renal/liver dysfunction, immunodeficiency conditions, or severe food allergies.

Inclusion Criteria

ADOS validated diagnoses of ASD

Questionnaires: RBS-2, KBIT, SRS, Rome III Version (QPGS- RIII), Ped QL, SSP

I am between 5 and 17 years old and have been diagnosed with autism.

Exclusion Criteria

I am capable of giving consent but choose not to.

You were born before 36 weeks of pregnancy.

Pregnancy: testing will be done on FMT day 0 for subjects with childbearing potential

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

4-6 weeks

1 visit (in-person)

Baseline Evaluation

Baseline symptom evaluation and medical exam to determine GI symptoms, including assessments using ADOS-2, RBS-R, SRS-2, and KBIT

Approximately 90 minutes

1 visit (in-person)

Treatment

Participants receive FMT or placebo through upper endoscopy

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including a phone call follow-up on Day 7

24 weeks

5 visits (including phone call follow-up)

What Are the Treatments Tested in This Trial?

Interventions

• FMT

Trial Overview The study tests if fecal microbiota transplantation (FMT)—transferring stool from a healthy donor to the intestines—helps kids with ASD. Participants are randomly chosen to receive either FMT or a saline placebo through an endoscopy into the jejunum.

How Is the Trial Designed?

Group I: Arm 2 Active Control 1 Intervention

Active Comparator: Donor Stool Transplant Arm 2 will get FMT (Fecal Microbial Transplant) with Healthy Donor Stool into the jejunum through upper endoscopy.

Group II: Arm 1 Placebo Group 1 Intervention

Placebo Comparator placebo into the jejunum through upper endoscopy.

Who Is Running the Clinical Trial?

Children's Hospital Los Angeles

Lead Sponsor

Trials

257

Recruited

5,075,000+

Published Research Related to This Trial

Cognitive Remediation Therapy (CRT) significantly improved executive functions and school performance in children and adolescents with Autism Spectrum Disorder (ASD), with 16 out of 25 participants completing an average of 22 sessions.

After CRT, participants showed notable enhancements in intellectual abilities, working memory, and specific aspects of cognitive flexibility, indicating that CRT is a promising intervention for addressing cognitive impairments associated with ASD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cognitive remediation therapy in autism spectrum disorder: Tunisian experience. Hajri, M., Abbes, Z., Ben Yahia, H., et al.[2020]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10017995/

Effect of fecal microbiota transplantation in children with ... Compared with the baseline, FMT significantly improved symptoms of autism in children with ASD in observational studies. However, rigorously designed randomized ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S2590097825000199

Faecal microbiota transplantation as a novel approach for ... Early clinical investigations, though limited, suggest that FMT may improve gastrointestinal health, core ASD symptoms, and comorbid anxiety. Mechanistically, ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06290258

Fecal Microbiota Transplantation for Patients With Autism ... This study aims to evaluate the efficacy of fecal microbiota transplantation on the gastrointestinal symptoms, autistic symptoms and emotional behavior ...

4. frontiersin.org frontiersin.org/journals/microbiomes/articles/10.3389/frmbi.2023.1222089/full

Fecal microbial transplantation as a novel therapeutic for ... Discussion: ASDs and their associated gastrointestinal symptoms are improved with FMT, potentially due to the engraftment of features of a ...

5. mdpi.com mdpi.com/2076-2607/13/6/1290

The Effects of Fecal Microbial Transplantation on ... FMT shows several advantages over probiotic supplementation in ASD: it has longer persistence than probiotics [31] and is more feasible, considering the food ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9762410/

Fecal Microbiota Transplantation in Autism Spectrum Disorder This review paper will therefore focus on FMT in the treatment of ASD, and FMT is effective in improving gastrointestinal and neurobehavioral symptoms in ...